DMD: Single CRISPR treatment provides long-term benefits in mice

Researchers have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease -- Duchenne muscular dystrophy (DMD) -- for more than a y..

Researchers have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease — Duchenne muscular dystrophy (DMD) — for more than a year in mice, despite observed immune responses and alternative gene editing outcomes.